Extended indication Treatment of older patients with treatment naive AML who are ineligible to receive chemotherapy.
Therapeutic value Possible added value
Registration phase Clinical trials

Product

Active substance Venetoclax / cytarabine
Domain Oncology and Hematology
Main indication Leukemia
Extended indication Treatment of older patients with treatment naive AML who are ineligible to receive chemotherapy.
Proprietary name Venclyxto
Manufacturer Abbvie
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Intermural (MSZ)
Additional remarks Apoptosis stimulant; proto-oncogene protein c-bcl-2 inhibitor.

Registration

Registration route Centralised (EMA)
Particularity Indication extension
Submission date 2020
Expected Registration 2021
Orphan drug Yes
Registration phase Clinical trials
Additional remarks Breakthrough-therapy designation granted by the FDA.

Therapeutic value

Current treatment options EMA: Treatment for AML is complex and depends on a number of factors including the extent of the disease, whether it has been treated before, and the patient’s age, symptoms and general state of health. At the time of designation, the main treatments for AML were chemotherapy (medicines to treat cancer) and haematopoietic (blood) stem-cell transplantation (a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow).
Therapeutic value Possible added value
Substantiation EMA: he sponsor has provided sufficient information to show that venetoclax might be of significant benefit for patients with AML because early studies show that, when used in combination with other medicines, it can produce a response in the disease in patients older than 65 years who cannot receive standard treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Frequency of administration 1 times a day
Dosage per administration 600 mg
Additional remarks Lijkt zeer veelbelovende combinatie; respons rates van 70-80%. Middel wordt binnenkort getest, in combinatie met intensieve chemotherapie, in HOVON studie.

Expected patient volume per year

Patient volume

20 - 120

Market share is generally not included unless otherwise stated.

References NKR; CBS
Additional remarks NKR 2015: 674. Clinical trial in patienten >65; CBS: percentage bevolking >65 jaar; 17,8%. Veel patiënten ontvangen chemotherapie. Om die reden verwachten we dat het realistisch patiëntvolume nog lager zal liggen.

Expected cost per patient per year

References Medicijnkosten.nl
Additional remarks Venetoclax € 56,58 per tablet van 100MG CYTARABINE € 33,25 per flacon OPLOSSING VOOR INFUSIE 100MG/ML FL 10ML.

Potential total cost per year

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No
References Clinicaltrials.gov, SPS.
Additional remarks Er lopen meerdere PhIII studies met Venetoclax maar niet in combinatie met cytarabine.

Other information

There is currently no futher information available.